Building on previous CMTA-funded success, new $450K investment accelerates research to treat CMT1A and CMT1B
The CMTA is thrilled to announce a significant investment of $450K into groundbreaking research targeting CMT1A and CMT1B, led Jordan VerPlank, Ph.D., at the Uniformed Services University of the Health Sciences.
This initiative marks a pivotal moment in our collective efforts to unravel the complexities of Charcot-Marie-Tooth disease (CMT) and to bring effective treatments for patients worldwide.
The CMTA-funded early work from the VerPlank lab showed that increasing cyclic guanosine monophosphate (cGMP) levels could improve myelination, nerve conduction, and motor coordination. The new research will investigate the mechanism by which boosting cGMP could mitigate neuropathy by facilitating the breakdown of disease-causing mutant proteins and preventing their harmful intracellular accumulation, in both laboratory studies and in animal models of CMT1A and 1B.
What sets this research apart? We're leveraging the concept of "drug repurposing" to expedite the translation of findings into clinical applications. By harnessing FDA-approved treatments, we aim to accelerate the development of targeted therapies for CMT subtypes.
This strategic investment underscores the CMTA's unwavering commitment to fostering collaboration and innovation in CMT research. Through our Strategy to Accelerate Research (STAR) initiative, we're driving forward transformative discoveries that hold the promise of improving patient outcomes.
Interested in contributing to this vital research or exploring partnership opportunities? Reach out to Dr Katherine Forsey, Ph.D., our chief research officer, to learn how you can partner with the CMTA to bring treatments to CMT and to improve the lives of those living with this disease.
Together, with Dr. VerPlank and others, we’re pushing the boundaries of scientific exploration and paving the way for innovative solutions for CMT.
Connect with Katherine Forsey, Ph.D., today: katherine@cmtausa.org